A global online study of haematopoietic stem cell transplantation in multiple sclerosis and other neurodegenerative disorders.

Abstract

The objective of this study was to understand the uptake of hemopoietic stem cell transplantation (HSCT) in neuroimmunological disorders like multiple sclerosis (MS). An independent University affiliated research organization conducted a global online survey of people having had HSCT, examining demographics, treatment protocol, and effectiveness. Of 271 participants, useful data were available in 223;women aged 35-54 accounted for 73.5%. Most had a household income greater than US$50,000, and the majority of participants were from Australia and the United States. Nearly94.6% of people sufferfrom MS. Most had their treatment in Russia (38.7%) and 78.1% had nonmyeloablative transplants. Nearly half of the participants spent between US$50,000 to US$74,999. There were 54.5% of neurologists who did not support their patients having HSCT. Around 85.5% of participants believed HSCT helped them manage their disease from weeks to years after transplantation,and treatment was recommended by 9.5% of participants. The average reduction in Expanded Disability Status Score after transplantation was 1.2 (95% CI: 0.97-1.41; N = 197; p < 0.01; t: 10.7, df: 196). Participants were supportive of HSCT despite the costs and would recommend it to others. The data suggest some benefit in minimizing disability in MS and provides justification for large randomized controlled trials.